Single 12 mg Dose of 5-MeO-DMT Reduces Depression Scores by 11.1 Points, Secures FDA Breakthrough Designation

NEW YORK, AMSTERDAM, and OXFORD, United Kingdom – The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to BPL-003, an intranasal formulation of 5-MeO-DMT benzoate developed by Beckley Psytech, for the treatment of adult patients with treatment-resistant depression (TRD). This significant milestone follows promising Phase 2b clinical trial results demonstrating rapid and durable antidepressant effects from a single dose. The designation aims to expedite the development and review process for drugs addressing serious conditions with potential for substantial improvement over existing therapies.

The Phase 2b core, blinded study involved 193 individuals with moderate-to-severe depression across 38 sites in six countries. Patients were administered either a 12 mg dose (n=73), an 8 mg dose (n=46), or a low-dose comparator of 0.3 mg (n=74), which served as a placebo control. The trial rigorously assessed the compound's efficacy and safety in a population where conventional treatments have failed.

Efficacy findings revealed that a single 12 mg dose significantly reduced depression scores by 11.1 points on the MADRS scale (0-60) at day 29 post-dosing, compared to a 5.8-point reduction with the 0.3 mg low-dose comparator. The 8 mg dose demonstrated equivalent efficacy, suggesting its potential for therapeutic benefit. Both therapeutic doses showed statistically significant improvements over the low-dose comparator, with effects beginning two days after treatment and peaking at 29 days, maintaining durable reductions of 10.2 and 10.8 points respectively.

Regarding safety, BPL-003 was well tolerated across all doses, with over 99% of adverse effects reported as mild or moderate, and no serious adverse events. While dose-related increases in nausea, headache, blood pressure, and anxiety were observed, the 8 mg dose was generally better tolerated than the 12 mg dose. Crucially, no suicide-related safety signals or concerns were identified, and most treated patients were ready for discharge within 90 minutes of treatment.

This Breakthrough Therapy designation, announced by atai Life Sciences and Beckley Psytech, underscores the urgent unmet need for new TRD treatments. The two companies previously announced a planned strategic combination to become a global leader in mental health therapies. Cosmo Feilding Mellen, CEO of Beckley Psytech, stated, "Receiving Breakthrough Therapy designation is a significant milestone which highlights the potential of BPL-003 to address the urgent unmet need of patients whose depression is not helped by existing therapies." This designation is expected to guide and expedite the pivotal Phase 3 clinical program, with trials anticipated to begin in the second quarter of 2026.